Clinical Trial: Pathophysiology of Dilated Cardiomyopathy

Study Status: Completed
Recruit Status: Unknown status
Study Type: Observational

Official Title: Using Novel Blood and Imaging Biomarkers to Better Understand the Pathophysiology of Paediatric Dilated Cardiomyopathy

Brief Summary:

This will be a cross-sectional, observational study.

Null hypothesis:

There is no difference in the amount of extracellular volume (ECV or scarring) in the hearts of patients with heart failure as compared to control subjects.

Heart failure occurs when the heart muscle has become too weak to work properly. It is associated with an increase in the amount of connective tissue (collagen) which replaces dead heart muscle cells (scarring). Currently a biopsy of the muscle is the only way to measure the amount of scarring. This is invasive and rarely done in children. Because of this, it is difficult to measure the amount of scarring in a particular patient or disease process, which is important for improving our understanding and treatment of the disease.

Cardiac magnetic resonance imaging (MRI) is a non-invasive imaging tool which is routinely used to look at areas of local scarring in heart muscle. Because the scarring is so widespread in paediatric patients, we have not been able to use this method previously. Now new imaging techniques allow us to look at widespread scarring but these have not yet been validated in children.

We plan to use late gadolinium enhancement (T1 mapping) to measure the amount of scarring in patients with heart failure (we have evidence that their heart biopsies show increased amounts of scar tissue) and children having MRI scans for other reasons. We will use measures of function including echocardiography and 6 minute walk test to compare to the amount of scarring. This will help us to know whether the amount of scarring will be clinically useful.

We will look at the amount of various proteins in the

Detailed Summary:

All patients will be under the care of Great Ormond Street Hospital.

Patient Groups:

There will be three main patient groups:

  1. Heart Failure Patients (MRI Clinically indicated)

  2. Heart Failure Patients (Voluntarily recruited from clinic)

    1. 8-16 years of age
    2. Non-GA only
  3. Control subjects (Clinically indicated brain MRI with contrast)

Patient recruitment

We will include Great Ormond Street patients in this trial who are known to the heart failure team because they have been diagnosed with DCM for at least 3 months. They will have an established diagnosis of heart failure and will be under regular follow up. Where possible, we will attempt to align the research study review date with that of the patient's regular annual review.

We will include patients who are to have a cardiac MRI scan with contrast for clinical reasons. These patients will be of any age and will have the scan under GA if appropriate, In addition, we will recruit patients from heart failure clinic who are not due to have scans for clinical reasons. These patients will be between 8-16 years to allow cooperation with the MRI scan protocol without the need for general anaesthetic (GA).

Age-matched control patients will be those having routine MRI with contrast for non-cardiac MRI scans. They will also have blood tests for cardiac biomarkers a
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Current Primary Outcome: Fibrosis [ Time Frame: 6 months ]

Higher fibrosis score (ECV) in heart failure patients in comparison to control subjects


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Biomarkers [ Time Frame: 6 months ]
    Significantly different pattern of blood biomarkers in heart failure patients as compared to control subjects
  • Disease Severity [ Time Frame: 6 months ]
    Significant correlation between fibrosis score on MRI or biomarker profile and clinical parameters (including 6 minute walk test and disease severity).


Original Secondary Outcome: Same as current

Information By: Great Ormond Street Hospital for Children NHS Foundation Trust

Dates:
Date Received: November 29, 2013
Date Started: January 2014
Date Completion:
Last Updated: November 29, 2013
Last Verified: November 2013